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Promising enzyme therapy shows Long-Term safety for rare fabry disease

NCT ID NCT03614234

Disease control Sponsor: Chiesi Farmaceutici S.p.A. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study looked at the long-term safety and effectiveness of a drug called pegunigalsidase alfa for adults with Fabry disease, a rare genetic condition. Participants received an infusion every four weeks for up to several years. The goal was to see if the drug is safe over time and helps control the disease.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

29 people

The number who actually took part.

Started

Nov 2018

Finished

Apr 2026

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Completion of study PB-102-F50. 2. The patient signs informed consent. 3. Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically accepted, effective contraception method. These include combined (estrogen- and progestogen-containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, or transdermal) supplemented with a barrier method (preferably male condom), progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, or implantable) supplemented with a barrier method (preferably male condom), intrauterine device (IUD), intrauterine hormone-releasing system (IUS), bilateral tubal occlusion, vasectomised partner, or sexual abstinence. Contraception should be used for 2 weeks after treatment termination. Exclusion Criteria: Presence of any medical, emotional, behavioral, or psychological condition that, in the judgment of the Investigator, would interfere with patient compliance with the requirements of the study.

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Conditions

The condition(s) this trial relates to.

Fabry disease proteinuria

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Fabry disease

Contacts and locations

Locations

  • Addenbrooke's Hospital

    Cambridge, CB2 0QQ, United Kingdom

  • Azienda Ospedaliera Universitaria "Federico II"

    Naples, Via Pansini, 80131, Italy

  • Emory University School of Medicine

    Atlanta, Georgia, 30307, United States

  • Fakultní poliklinika Všeobecné fakultní nemocnice v Praze

    Prague, 128 08, Czechia

  • Helse Bergen HF Haukeland Universitetssykehus

    Bergen, 5021, Norway

  • Infusion Associates

    Grand Rapids, Michigan, 49525, United States

  • Medical Endocrinology PE 2132, Rigshospitalet

    Copenhagen, 2100, Denmark

  • O & O Alpan

    Fairfax, Virginia, 22030, United States

  • Renal Disease Research Institute, LLC

    Dallas, Texas, 75235, United States

  • The Royal Free Hospital

    London, United Kingdom

  • UAB Medicine

    Birmingham, Alabama, 35233, United States

  • UZ Antwerpen

    Edegem, 2650, Belgium

  • University of Iowa Hospitals and Clinica

    Iowa City, Iowa, 52242, United States

  • University of Utah Hospitals & Clinics

    Salt Lake City, Utah, 84112, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.